Navigation Links
Repeated hUCB injections may improve prognosis of children with deadly inherited disorder
Date:3/28/2014

Putnam Valley, NY. (Mar. 28 2014) New insight has been gained into treating an inherited disorder that creates serious neurological and behavioral disabilities in children and usually leads to death in the teen years.

In a recent study into the effects of human umbilical cord blood mononuclear cells (hUCB MNCs) when they are injected to counter the symptoms and progression of Sanfilippo syndrome type III B (MPS III B), researchers found that repeated injections into laboratory mice modeled with the disorder had clear benefits for the mice receiving multiple injections over control groups that received single injections of either a high or low dose of cells.

The study will be published in a future issue of Cell Transplantation but is currently freely available on-line as an unedited early e-pub at: http://www.ingentaconnect.com/content/cog/ct/pre-prints/content-ct1121willing.

MPS III B results from a genetically programmed deficit of the Naglu enzyme. The deficit creates a build-up of heparan sulfate - a complex carbohydrate - that accumulates in lysosomes, cells that are responsible for waste disposal. With MPS III B, accumulations of heparin sulfate in the tissues are not eliminated and the accumulation causes damage to multiple organs, including the brain.

"Cell therapy has recently received attention as a potential treatment for lysosomal storage diseases," said study lead author Dr. Allison E. Willing, of the Center of Excellence for Aging and Brain Repair in the Morsani College of Medicine at the University of South Florida. "We have previously shown that a single hUCB injection into the cerebral ventricle of pre-symptomatic mice, or intravenous cell delivery at different disease stages, had a beneficial effect on the enzyme deficient mice. In the current study, we examined whether administering repeated doses of hUCB MNCs would have a greater effect than a single dose and help to prevent progressive neurodegeneration."

Using three groups of mice modeled with Naglu deficiency by knocking out the Naglu enzyme, the researchers injected one group with repeated doses of hUCB MNCs over a six month period. They administered single doses - either high or low doses - to two other groups of similarly modeled mice. The group that had repeated hUCB MNC doses demonstrated a variety of favorable benefits.

To determine the benefits of repeated hUCB MNC injections, the researchers measured several behavioral and clinical outcomes before and after six months of treatment. These included anxiety, levels of heparin sulfate accumulation, and subsequent pathology in various anatomical brain locations.

"Repeated injections of hUCB MNCs produced the greatest neuroprotection," stated study co-lead author Dr. Svitlana Garbuzova-Davis, of the Center of Excellence for Aging and Brain Repair in the Morsani College of Medicine at the University of South Florida. "Hippocampal structural architecture remained intact in the repeated dose-treated mice as compared to the other groups. Also, there was restoration of the dendritic tree in the group receiving repeated doses. We also saw a striking reduction in microgliosis and microglial activation after hUCB MNC treatment."

The researchers speculated that administering hUCB MNCs may decrease neuropathy through modulation of inflammatory and immune processes as the hUCB MNCs produced numerous neurotrophic and growth factors.

"We demonstrated that hUCB MNCs were particularly effective at modulating anxiety in the Naglu knockout mice,"concluded Paul R. Sanberg, distinguished professor at USF and principal investigator of the Children's Medical Research Foundation funded project. "Our results suggest that repeated administrations of hUCB MNCs produce greater amelioration of the underlying disease pathology. However, further studies will be necessary to determine if this treatment regimen can slow the progression of the disease, increase survival while minimizing symptoms, and determine whether improved outcomes are a function of enzyme administration, decreased inflammation, or both."

"This study highlights the benefits of using multiple injections rather than a single injection to treat Sanfilippo syndrome type III B" said Dr. John Sladek, Cell Transplantation section editor and professor of neurology and pediatrics at the University of Colorado School of Medicine. "This use of multiple treatments may be applicable to other neurodegenerative disorders, as already suggested by animal studies published by the same group for the treatment of amyotrophic lateral sclerosis."
'/>"/>

Contact: Robert Miranda
cogcomm@aol.com
Cell Transplantation Center of Excellence for Aging and Brain Repair
Source:Eurekalert

Related medicine news :

1. Repeated exposure to traumatic images may be harmful to health
2. Study: Repeated surgeries appear to extend life of patients with deadliest of brain cancers
3. Breast cancer risk estimates increased with repeated prior ct and nuclear imaging
4. No need for routine repeated CT scans after mild head trauma, reports neurosurgery
5. Facial dog bites in children may require repeated plastic surgery
6. Necrosis after cortisone injections
7. Reused Vials, Unsafe Injections Threatening Patients: CDC
8. US Vitamin Injections Looks Into B12 Shots for Fitness in New Article
9. US HCG Injections Posts New Article about the Spartacus Workout
10. US HCG Injections Posts Inspirational HCG Diet Customer Success Story
11. hCGTreatments / Diet Doc hCG Diets & Weight Loss Plans Announces New Alternative to Stomach Botox Injections
Post Your Comments:
*Name:
*Comment:
*Email:
(Date:10/13/2017)... ... October 13, 2017 , ... Global Healthcare Management’s 4th Annual Kids Fun Run ... This free event, sponsored by Global Healthcare Management’s CEO, Jon Letko, is aimed ... geared towards children of all ages; it is a non-competitive, non-timed event, which is ...
(Date:10/13/2017)... ... October 13, 2017 , ... “The Journey: From the Mountains ... possible to save lost souls in the Philippines. “The Journey: From the Mountains to ... dedicated teacher of the Bible. She has taught all ages and currently teaches a ...
(Date:10/12/2017)... ... 12, 2017 , ... Planet Fitness, one of the largest and fastest growing ... open a flagship location in Covington, LA at 401 N. U.S. Highway 190, in ... to Office Depot in the Holiday Square shopping center. Its location allows it to ...
(Date:10/12/2017)... ... October 12, 2017 , ... Asante, a nationally recognized ... have expanded their existing home health joint venture through an agreement, effective October ... a joint venture home health company with Asante, delivering clinically integrated care, for ...
(Date:10/12/2017)... ... October 12, 2017 , ... Leading pediatric oncology experts at Children’s ... the 49th Congress of the International Society of Paediatric Oncology (SIOP) Oct. ... Center for Cancer and Blood Disorders at Children’s National, and Stephen P. Hunger, ...
Breaking Medicine News(10 mins):
(Date:10/2/2017)... 2017 The Rebound mobile app is poised to ... the tide of prescription drug addiction. The app empowers users ... and stepping down their dosage in a safe, controlled manner ... 2017; the first 100,000 people to sign up will enjoy ... ...
(Date:9/27/2017)... Sept. 27, 2017  Commended for their devotion to personalized ... Ranked as number one in the South Florida Business Journal,s ... Inc. 5000 yearly list, the national specialty pharmacy has found ... Bardisa will soon be honored by SFBJ as the ... Set to receive his award in October, Bardisa ...
(Date:9/25/2017)... , Sept. 25, 2017  EpiVax, Inc., ... vaccine design, and immune-engineering today announced the launch ... the development of personalized therapeutic cancer vaccines. EpiVax ... provided exclusive access to enabling technologies to the ... MBA will lead EpiVax Oncology as Chief Executive ...
Breaking Medicine Technology: